EU/3/11/923

Orphan designation

On 27 October 2011, orphan designation (EU/3/11/923) was granted by the European Commission to Orphix Consulting GmbH, Germany, for L-cysteine, L-leucyl-L-alpha-glutamyl-L-alpha-glutamyl-L-lysyl-L-lysylglycyl-L-asparaginyl-L-tyrosyl-L-valyl-L-valyl-L-threonyl-L-alpha-aspartyl-L-histidyl-S-[1-[(4-carboxycyclohexyl)methyl]-2,5-dioxo-3-pyrrolidinyl]-complex with keyhole limpet haemocyanin (also known as rindopepimut) for the treatment of glioma.

Glioma is a type of brain tumour that affects the ‘glial’ cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain. Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About a fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.

Glioma is a long-term debilitating and life-threatening disease because of severe damage to the brain which is associated with poor long-term survival.

At the time of designation, glioma affected approximately 1.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 61,000 people, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments for glioma included surgery and radiotherapy (treatment with radiation), as well as chemotherapy (medicines to treat cancer) to improve survival. Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull and medicines to prevent seizures.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with glioma because it works in a different way to existing treatments and might be used in combination with existing methods to improve the treatment of patients. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The medicine is a ‘cancer vaccine’. It is expected to work by activating the patient’s immune system (the body’s natural defences) so that it attacks and destroys the tumour cells of the glioma.

This vaccine is based on a peptide (a short chain of amino acids) which is specific to a variant of the epidermal growth factor receptor known as EGFRvIII which is involved in the growth and spread of cancer cells and is found on the tumour cells in some glioma patients. To prepare this vaccine, the peptide is produced in a laboratory. It is attached to a carrier, called ‘keyhole limpet haemocyanin’ (a protein prepared from a sea snail), which acts as an immune stimulant to enhance the response of the body to the EGFRvIII peptide in the vaccine. When this vaccine is given, the patient’s immune system is expected to recognise the EGFRvIII peptide as ‘foreign’. This is expected to stimulate an immune response against the glioma cells containing this peptide, resulting in the immune system attacking and destroying these cells, but leaving other cells in the body unharmed.

the existence of alternative methods of diagnosis, prevention or treatment;

either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.